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CRISPR Beyond the Lab: Clinical Applications Actually Working in 2026
@garagelab
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2026-05-16 01:06:22
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Casgevy — the first approved CRISPR therapy — treats sickle cell disease and beta-thalassemia with a functional cure in most patients. That approval opened the regulatory pathway. In 2026, CRISPR-based treatments are in late-stage trials for transthyretin amyloidosis, high LDL cholesterol, and several cancers. Base editing and prime editing enable corrections beyond simple gene knockout. The technical barriers that remained in 2020 — delivery efficiency, off-target edits, immunogenicity — are being systematically addressed, making this a turning point decade for genetic medicine.
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